Jazz Pharmaceuticals Announces First Patient Enrolled in EMERGE-201 Phase 2 Basket Trial Evaluating Zepzelca® (lurbinectedin) Monotherapy in Patients with Select Advanced or Metastatic Solid Tumors
Open-label basket trial will assess the safety and efficacy of Zepzelca in patients with advanced urothelial carcinoma, large cell neuroendocrine carcinoma of the lung, or homologous recombination deficient tumors
"Even with the rapid advancements in medical technology and care delivery across many tumor types over the last decade, there still remain many patients who continue to face unmet needs and experience a high burden of morbidity and mortality," said
"This trial initiation is an exciting milestone for Zepzelca's clinical development program, as we seek to evaluate its clinical utility beyond treating small cell lung cancer," said
EMERGE-201 is a Phase 2, multicenter, open-label trial designed to assess the safety and efficacy of Zepzelca as a monotherapy in three cohorts of patients with solid tumors, who will receive 3.2 mg/m2 doses of Zepzelca intravenously on day one of an every-three-week dosing cycle, until confirmed disease progression. The three cohorts are: patients with advanced urothelial carcinoma, large cell neuroendocrine carcinoma of the lung, or HRD tumors who have progressed on a platinum-containing regimen.
The primary objective is to determine Zepzelca's ability to improve patient outcomes, as measured by ORR. Key secondary endpoints include investigator-assessed progression free survival, time-to-response, duration of response, and disease control rate as assessed by RECIST, as well as overall survival in participants treated with Zepzelca. The trial is sponsored and is being conducted by
Approximately 20 sites in the
Zepzelca is an alkylating drug that binds guanine residues within DNA. This triggers a cascade of events that can affect the activity of DNA binding proteins, including some transcription factors, and DNA repair pathways, resulting in disruption of the cell cycle and eventual cell death.
The FDA approved Zepzelca under accelerated approval in June 2020 for the treatment of adult patients with metastatic SCLC with disease progression on or after platinum-based chemotherapy. The approval is based on overall response rate (ORR) and duration of response demonstrated in an open-label, monotherapy clinical study. In
Zepzelca is a prescription medicine used to treat adults with small cell lung cancer that has spread to other parts of the body (metastatic) and who have received treatment with chemotherapy that contains platinum, and it did not work or is no longer working. Zepzelca is approved based on response rate and how long the response lasted. Additional studies will further evaluate the benefit of Zepzelca for this use. Zepzelca is not approved as part of a combination therapy or as a first-line maintenance treatment for patients with extensive-stage small cell lung cancer.
Before receiving ZEPZELCA, tell your healthcare provider about all of your medical conditions, including if you:
- have liver or kidney problems.
- are pregnant or plan to become pregnant. ZEPZELCA can harm your unborn baby.
Females who are able to become pregnant:
- Your healthcare provider should do a pregnancy test before you start treatment with ZEPZELCA.
- You should use effective birth control (contraception) during treatment with and for 6 months after your final dose of ZEPZELCA.
- Tell your healthcare provider right away if you become pregnant or think that you are pregnant during treatment with ZEPZELCA.
Males with female partners who are able to become pregnant should use effective birth control during treatment with and for 4 months after your final dose of ZEPZELCA.
Females who are breastfeeding or plan to breastfeed. It is not known if ZEPZELCA passes into your breastmilk. Do not breastfeed during treatment with ZEPZELCA and for 2 weeks after your final dose of ZEPZELCA. Talk to your healthcare provider about the best way to feed your baby during treatment with ZEPZELCA.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Certain other medicines may affect how ZEPZELCA works.
Avoid eating or drinking grapefruit, or products that contain grapefruit juice during treatment with ZEPZELCA.
ZEPZELCA can cause serious side effects, including:
- Low blood cell counts. Low blood counts including low neutrophil counts (neutropenia) and low platelet counts (thrombocytopenia) are common with ZEPZELCA, and can also be severe. Some people with low white blood cell counts may get fever, or an infection throughout the body (sepsis), that can cause death. Your healthcare provider should do blood tests before you receive each treatment with ZEPZELCA to check your blood cell counts.
Tell your healthcare provider right away if you develop:
- fever or any other signs of infection
- unusual bruising or bleeding
- pale colored skin
- Liver problems. Increased liver function tests are common with ZEPZELCA, and can also be severe. Your healthcare provider should do blood tests to check your liver function before you start and during treatment with ZEPZELCA.
Tell your healthcare provider right away if you develop symptoms of liver problems including:
- loss of appetite
- nausea or vomiting
- pain on the right side of your stomach area (abdomen)
- Your healthcare provider may temporarily stop treatment, lower your dose, or permanently stop ZEPZELCA if you develop low blood cell counts or liver problems during treatment with ZEPZELCA.
The most common side effects of ZEPZELCA include:
- low white and red blood cell counts
- increased kidney function blood test (creatinine)
- increased liver function blood tests
- increased blood sugar (glucose)
- decreased appetite
- muscle and joint (musculoskeletal) pain
- low level of albumin in the blood
- trouble breathing
- low levels of sodium and magnesium in the blood
These are not all of the possible side effects of ZEPZELCA.
Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to
More information about Zepzelca, including Full Prescribing Information and Patient Information, is available here.
ZEPZELCA is a trademark of
Urothelial carcinoma (UC) is the fourth most common tumor in
Large cell neuroendocrine carcinoma (LCNEC) is a rare type of cancer that can occur in the lungs and colon. There is no standard of care for LCNEC in lung cancer due to limited data on its pathology and clinical trials, making it a condition with great unmet needs in treatment and management.
Homologous recombination deficiency (HRD) occurs across a variety of solid tumors including endometrial, biliary tract, urothelial, breast, pancreatic, gastric and esophageal. HRD hinders the body's essential mechanism that repairs damaged DNA in cells. HRD tumors are seen in almost a fourth (17.4%) of tumors across 21 cancer types.
This press release contains forward-looking statements, including, but not limited to, statements related to
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1 Kim M, Jeong CW, Kwak C, et al. Are urothelial carcinomas of the upper urinary tract a distinct entity from urothelial carcinomas of the urinary bladder? Behavior of urothelial carcinoma after radical surgery with respect to anatomical location: a case control study. BMC Cancer 15, 149 (2015).
2 Tanaka M, Sonpavde G. Diagnosis and management of urothelial carcinoma of the bladder. Postgrad Med. 2011 May;123(3):43-55
3 Lindsay CR, Shaw EC, Moore DA, et al. Large cell neuroendocrine lung carcinoma: Consensus statement from
4 Heeke AL, Pishvaian MJ, Lynce F, et al. Prevalence of homologous recombination–related gene mutations across multiple cancer types. JCO Precis Oncol. 2018;2018:PO.17.00286.
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